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    EMA Consults on Regulatory Requirements for Chronic Liver Disease Drugs

    The European Medicines Agency (EMA) on Thursday launched a public consultation to gather input on a future reflection paper discussing the regulatory requirements for developing drugs to treat chronic non-infectious liver diseases. According to the agency, there is an unmet medical need for drugs to treat chronic non-infectious liver diseases such as non-alcoholic steatohepatitis (NASH), primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC). And wh...
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    FDA Approves First Treatment for a Form of Batten Disease

    The US Food and Drug Administration (FDA) on Thursday approved the first-ever treatment for a form of the rare pediatric Batten disease, bringing Biomarin its second lucrative priority review voucher (PRV). FDA approved Brineura (cerliponase alfa) to slow the loss of walking ability in symptomatic pediatric patients three years old and older with late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency. ...
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    GAO: Drugmakers Want More Clarity on Antibiotic Incentives

    Despite some improvements, drugmakers say the US Food and Drug Administration (FDA) has not fully clarified its expectations for developing new antibiotic drugs or detailed how to access new incentives, according to a new report by the Government Accountability Office (GAO). Specifically, GAO says that FDA has issued 14 updated or new guidances focused on antibiotics since the Generating Antibiotic Incentives Now (GAIN) Act was passed as part of the Food and Drug Admi...
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    FDA Finalizes Guidance on Tropical Disease PRVs, Offering a Number of Clarifications

    The US Food and Drug Administration (FDA) on Wednesday finalized guidance on tropical disease priority review vouchers (PRVs), clarifying when a voucher can be used, whether drugmakers are “guaranteed” a six-month review when using a voucher and whether FDA can remove a tropical disease from the list of considered diseases (it can’t). This finalization of the 2008 draft guidance includes 25 questions and answers and includes the following substantive changes based on pu...
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    Priority Review Voucher Fees to Decline in FY 2017

    The US Food and Drug Administration (FDA) on Thursday unveiled the new user fee rates for the tropical disease and rare pediatric disease priority review voucher (PRV) programs. The additional fees necessary to use the vouchers for both programs are set to decline by about $20,000 when compared to last year. Tropical Disease Priority Review Voucher User Fee Year Voucher Fee FY 2011 $4,582,000 FY 2012 $5,280,000 FY 2013 $3...
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    Congress Signs Off on Short-Term Extension to Rare Pediatric PRV Program

    The House late Tuesday followed its Senate counterparts and sent to President Barack Obama a re-authorization of the rare pediatric disease priority review voucher (PRV) program that will last through the end of December. A spokeswoman for Sen. Bob Casey (D-PA), a sponsor of the bill, told Focus that between now and 30 December, Congress will discuss ways to amend the program and extend it again. “Our policy changes are in S. 1878, which is headed to the president, so...
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    Industry Weighs in on FDA Draft Guidance on Infectious Disease NGS-Based Diagnostics

    Diagnostic companies Illumina, Roche, AstraZeneca and others are offering a peek inside how they view next-generation sequencing (NGS) diagnostic devices, with the release of 20 comments on recent US Food and Drug Administration (FDA) draft guidance. "Next generation sequencing has the capability to replace previous methods with a single approach to accomplish what might have required several different tests in the past," FDA explains. "In contrast to human sequencing di...
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    EMA and FDA Set Up New Working Group on Rare Diseases

    The new collaboration between the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) announced Monday will improve the sharing of information on various aspects of the development and scientific evaluation of medicines for rare diseases. According to EMA, rare diseases affect 30 million people in the EU and approximately the same number in the US, though each disease individually concerns a limited number of patients. And since only a limited n...
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    EMA Official: Data Sharing and Collaboration Can Fill Scientific Gaps in Alzheimer's

    Speaking at a panel at RAPS' 2016 Convergence on Monday, Enrica Alteri, head of human medicines research and development support at the European Medicines Agency (EMA) called on drugmakers to share data to close the gaps in Alzheimer's disease (AD) research. According to Alteri, doing so could help the scientific community not only learn from past failures in Alzheimer's research, but enable the development and validation of new targets. Scientific Gaps "There are gaps...
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    European Regulatory Roundup: EMA Begins Review of First Herceptin Biosimilar (1 September 2016)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. EUnetHTA Nears Start of Third Phase of HTA Cooperation Agenda The European network for Health Technology Assessment (EUnetHTA) is on the cusp of entering the third phase of its agenda. Members of the European Commission and the Dutch National Health Care Institute began a 30-day countdown to the official start of the program by signing a grant agreement.  When the of...
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    FDA Issues Form 483 for Alexion’s Rhode Island Site

    Rare disease drugmaker Alexion disclosed late Monday in an SEC filing that the US Food and Drug Administration (FDA) recently completed a routine current Good Manufacturing Practice (GMP) inspection of the company’s Smithfield, Rhode Island manufacturing facility and issued a Form 483 with three observations. “These observations are inspectional, and do not represent a final FDA determination of compliance. The observations pertain to: completion and closure of certain ...
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    European Regulatory Roundup: EMA to Revise Trial Guidance on Hemophilia, Crohn’s, Ulcerative Colitis, Diabetes and Bipolar Disorder (4 August 2016)

    Welcome to our European Regulatory Roundup, our weekly overview of the top EU regulatory news. Boom in Hemophilia R&D Prompts EMA to act to Prevent Enrollment Bottleneck The European Medicines Agency (EMA) is planning to revise its guideline on development of drugs to treat hemophilia to cut the risk of patient enrollment bottlenecks slowing progress. EMA began the process after becoming concerned that the minimum data requirements of its current guideline are i...